Friday, 30 November 2012

The BigPharma Alternative

#MSBlog: Is there an alternative to BigPharma? Help make it happen!

Is there an alternative to Big Pharma for developing new drugs for MS?

With the current level of risk associated with drug development and the level of regulatory red-tape it seems unlikely. Even Big Pharma themselves are having second thoughts about the current paradigm and trying new things. Many companies have closed down their neuroscience research centres in Europe and are partnering with academic institutions to reduce the costs of preclinical drug development. Ten of the world’s biggest drug makers have launched Transcelerate Biopharma to reduce costs associated with the complexity of drug development. Transcelerate Biopharma is focusing on areas where clinical drug development is wasteful and duplicates procedures like training clinicians, running websites or data portals, or creating procedures to document the benefits and side effects of new medicines. Interestingly, Transcelerate Biopharma will be a non-profit. The ten companies that are supporting it are:

Abbott

AstraZeneca

Boehringer-Ingelheim

Bristol-Myers Squibb

Eli Lilly and Company

GlaxoSmithKline

Johnson & Johnson

Pfizer

Genentech-Roche

Sanofi

The aims of Transcelerate Biopharma are admirable, but they still underpin BigPharmas' main aim of launching novel drugs that are big earners. The problem we are having in academia is the repurposing of drugs, or developing drugs that BigPharma have discarded. Take Simvastatin for example; how is Dr Chataway and his colleagues, who have a positive phase 2 neuroprotective study, going to take this forward when Simvastatin is off patent? Merck-Serono stopped the development of oral cladribine for MS, despite it already being licensed in Russia and Australia. Can we, the MS community resuscitate cladribine? The cladribine results are very good and in my opinion would make a big difference to the treatment of RRMS and potentially progressive MS. How do we get cladribine back on the table?

We need an alternative system to one BigPharma uses. Regulators and politicians need to acknowledge this; in particular in this age of austerity when drug budgets are soaring. The current pharma model is broken and the costs associated with it are too high to be sustainable in the long-term. I have been asking you the readers of this blog, in particular those who are always bashing BigPharma, for alternatives for over 2 years. Some of you have suggested a new Tax or levy on BigPharma drugs to pay for non-profit drug development. This is unlikely to work if the regulatory red-tape remains so complex and expensive. In addition, the skill set resides in industry. Taxation as a way of paying for new initiatives seldom works; it is inefficient. Personally, I would prefer a market-driven system.

As a start we need to get regulators and politicians to lower the bar for non-profit or academic drug development. They should create incentives in a similar way they have done for orphan drug development. For example, allow the design of adaptive phase 3 efficacy programmes to be done at the same time the drug is given a provisional license. This will allow the necessary data to be collected at the same time the drug is being marketed under controlled conditions. For example, the EMA and FDA have concerns about the long-term safety of oral cladribine. Why not give the drug a provisional license and allow the drug to be sold on condition that every MSer who goes on the drug is registered in a well-controlled pharmacovigilance study to assess the risk of the drug. This study would need a control group to compare the relative risks; the control group could be subjects going onto interferon-beta or glatiramer acetate. Instead the EMA and FDA have thrown the baby out with the bathwater and MSers are being denied a potentially very effective drug. We need to change this!

I therefore propose starting a new organisation to lobby our politicians to change the paradigm and create an alternative to BigPharma to develop drugs for MS. The working model will be a non-profit, or social entrepreneurship, with the aim of getting legislation enacted similar to that for orphan-diseases; the primary objective to aid academic and non-profit drug development. The aim is to get drugs - which are off-patent or don’t have the necessary IP life-span for BigPharma to recoup development costs and make a profit - to market at an earlier stage under a provisional license, whilst the data is being collected for a definitive or full license.

This organisation needs a name, seed money and help from politicians or stakeholders. If you want to be part of this initiative please register your interest below and any suggestions you may have to help. The organisation will need a leader, administrators, secretaries, etc. Volunteers are most welcome.

Not sure MD, for some people that would throw up connections to big pharma as is, me thinks a total re brand. Also Tony Fonda makes a good point below, MRF have been adopting this model for sometime ? and not wishing to bait the bear ;-) i think they will be first in the race to unlock the potential of Stem Cells. Could you collaborate ?or are there too many coi's?Regards as always.

I can collaborate with any one, CoI is for your benefit to determine if you think we are giving reasonable opinions of our own, but stem cells is a very different issue from repurposing an exsisitng drug that is safe for human use and is generic.

Not really. The chances of anything coming out of the MRF that will make a meaningful contribution to MS in the near future is in my opinion some way off. We need to push for the repurposing of existing drugs that have proven neuroprotective potential for MS.You can't remyelinate a nerve that's already dead. Once we can stop them dying then we can think about myelin repair.

This is pertinent to the article. We desperately need a way of speeding up the process, particularly for drugs which have proven neuroprotective potential and can be re-purposed for MS.Give us your money Pharma!http://www.guardian.co.uk/business/2012/nov/27/new-uses-old-drugs-business

Very interesting discussion here - spent the last couple of hours thinking about a couple of ideas:what business model can leverage on non-patented drugs while pushing testing and trial costs to the public service/academia/NGO market?.

Probably a business that can manufacture cheaply, keep a tap on profit margins, strong worldwide distribution network, 0 marketing (not needed), works hand-in-hand with unis without actually funding any trials.

I like where Tony is going. I do think you need marketing (or a good communication strategy), however, to make clear to investors (academic, crowd sourced, government, or otherwise) how this model differs from traditional big pharma. And why they should participate.

Furthermore, suppose you are successful with a trial. You probably need marketing to some degree to combat misperceptions that might arise--intentionally or not. But that's way down the road.

Dang it. Now I've got excited about this. Are you interested in help from the US?

Yes, I would champion and I would lobby too. I think we'd have a compelling message that we could bring down overall health costs by coming up with a system for funding research into less expensive drugs--for MS and maybe many other diseases.

Since I'm in the US, we'd have to be careful to structure it so that it in no way smells like s-o-c-i-a-l-i-s-m. But I think it can be done.

Hey Cat stay excited! I'm sure help from all over the world will be appreciated, speaking on my own behalf, naturally. I guess we wait for Prof G to get his feet back on the ground after his travels and get his head together with the rest of his team.

If you guys want to end MS, figure out what MS actually is and most importantly, share your finding with the World. Today, MS is a perfect storm in which huge dollars are made preserving the suffering of the most desperate in a strictly controlled economic context. Shatter that context, by again, figuring out what MS actually is, and you will solve the problem you are genuinely trying to solve... ending the suffering of those with this merciless condition. Accept that in present context, 'any' compound of potential benefit to those with MS will be unapologetically Lemtrada'd in the nexus between patient desperation, clinical ignorance, regulatory impotence and financial greed.

Not quite on the topic of this post, but perhaps of interest. The economist Tyler Cowen has some comments on Big Pharma and Ben Goldacre at http://marginalrevolution.com/marginalrevolution/2012/11/bad-pharma-by-ben-goldacre.html.

"Could he not have called the book Not Nearly as Good as it Could be Pharma: How Corruption is Diminishing One of Our Great Benefactors? Admittedly that does not roll off the tongue as nicely.

"Or how about Slow Pharma: How to Get the New Drug Pipeline Up and Running Again?

"Goldacre’s policy recommendations would in general raise the costs of research and development, although they would likely improve the accuracy of research results and reduce over-prescription and overuse of drugs. It is quite possible they would lower the rate of return to pharmaceutical innovation, likely I would say. These trade-offs are neglected..."

I agree; not everything is wrong with BigPharma. They are still the most innovative show in town and we need their deep pockets to develop new and novel treatments. Anybody who thinks we can do this without them should try! The hurdles, stumbling blocks and volume of regulatory red tape is enough to make a grown man cry. One pharma exec told me recently that even the industry is having difficulties wading through the regulatory treacle.

You have just given me an idea; I am going to contact Ben Goldacre and invite to join our movement.

G &MD, I would love it if you can initiate an "intellectual"/thematic posit on this topic:

http://www.technologyreview.com/news/508186/gene-therapy-on-the-mend/

I understand that 60 defective genes or so have already been identified in MSers. What needs to be done to address this matter? what effort is being deployed around the world this specifically? What is barts doing in this space? [...]

I would not necessarily call them defective but I think that they are normal genes that in a particular combination predisose you to MS, but in other contexts these genes will help you fight infection.

Remember if you have an identical twin there is 70% chance of them not developing MS.

The individual genes make a little part of an effect compared to the obvious single gene mutations that cause some disease. 50 variants have been found and another 60 variants are suspected, but there will be invariant genes that are vital to the process so more will be involved in susceptibility. Changing them all would be a hard task.

In terms of gene therapy we are engineering stem cells to produce more myelinating cells, clinical at the moment nothing

PML Risk Infographic

Holistic Management of MS ver. 7.0

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